Vironexis Biotherapeutics has unveiled with $26 million and a clinical-trial-ready gene therapy that has already nabbed FDA fast track and rare pediatric disease tags.
The San Diego-based biotech’s seed financing was led by Drive Capital and Future Ventures with participation from Moonshots Capital and Capital Factory. The investors are backing Vironexis’ mission to develop off-the-shelf, single-dose gene therapies that treat cancer more effectively, more safely and with more durability.
Vironexis currently touts at least 10 preclinical pipeline candidates—including one cancer vaccine—that mostly take aim at blood-based cancers and solid tumor metastasis prevention. The programs are built from the biotech’s AAV gene therapy platform, dubbed TransJoin. The tech aims to bridge T cells and tumor cells for long-term, steady serum levels of the therapy and, subsequently, consistent T-cell-mediated tumor cell killing. Furthermore, the platform’s low-dose AAV delivery is designed to reduce toxicity and adverse events.
“We were drawn not only to the novelty of the TransJoin technology but also to its broad applicability, spanning treatments for blood-based cancers, solid tumor metastasis prevention, and cancer vaccines, as well as immune disorders," Steve Jurvetson, co-founder of Future Ventures, said in a Sept. 12 company release, adding that the platform's versatility "is truly standout."
The company’s lead gene therapy candidate is VNX-101, which has received FDA clearance to enter in-human testing, according to the company release. VNX-101 will be studied as a treatment for patients with CD19+ acute lymphoblastic leukemia, with Vironexis expecting to start enrollment for a phase 1/2 trial in the last quarter of this year.
The gene therapy has landed both fast track and rare pediatric disease designations from the FDA, the former of which is a process designed to speed up drug development and review, while the latter is a voucher aimed at incentivizing rare pediatric disease drug development. The FDA is planning on sunsetting the rare pediatric designation priority review voucher program after Sept. 30.
The VNX-101 study will be the first clinical trial of an AAV-delivered cancer immunotherapy, according to Vironexis.
“Our novel technology builds on the power of T cell immunotherapy while overcoming key shortcomings and challenges of existing approaches such as CAR-T and bispecific antibodies,” Vironexis CEO and co-founder Samit Varma said in the company release. “We believe we have the opportunity to dramatically improve upon the safety, efficacy and durability of these drug classes, while streamlining manufacturing and significantly lessening the burden of treatment for patients.”
The biotech, which launched three years ago, licensed the platform science from Nationwide Children’s Hospital in Columbus, Ohio. Research on the TransJoin platform was led by Timothy Cripe, M.D., Ph.D., the Nationwide hospital’s chief of pediatric hematology/oncology/bone and marrow transplant, and one of Vironexis’ co-founders.
“Recognizing the pivotal impact of AAV delivery for the treatment of rare diseases, we believed its unique ability to enable long-term, continuous expression of a therapeutic protein could be the missing link to overcome the myriad challenges associated with first-generation T cell immunotherapies like CAR-T,” said Cripe in the release, adding that the biotech has demonstrated the approach’s potential in preclinical models.
Cripe and gene therapy company entrepreneur Varma are joined by the biotech’s third co-founder, Brian Kaspar, Ph.D., a scientist-entrepreneur who founded AveXis, which was bought by Novartis in 2020, and pioneered the AAV gene therapy Zolgensma.
Vironexis’ second program, VNX-202, is designed to prevent metastatic HER2+ cancer, including breast cancer and other tumor types. The company hopes the asset will enter clinical testing next year.
The biotech’s other pipeline products include a cancer vaccine for GP350+ nasopharyngeal cancer and an investigational treatment designed to treat CD19+ systemic lupus erythematosus, a program Vironexis says it plans to partner for future development.